AAV-based gene therapies hold promise for treating disease, but their long-lasting gene expression limits their use in regenerative medicine. A study now presents DreAM, a drug-inducible AAV system that enables tunable tissue-specific gene activation via the splicing modulator risdiplam, and its potential application in myocardial infarction.
D'Uva, G. (2025). A tunable gene therapy for heart regeneration. NATURE CARDIOVASCULAR RESEARCH, 4(7), 794-796 [10.1038/s44161-025-00666-6].
A tunable gene therapy for heart regeneration
D'Uva G.
Primo
2025
Abstract
AAV-based gene therapies hold promise for treating disease, but their long-lasting gene expression limits their use in regenerative medicine. A study now presents DreAM, a drug-inducible AAV system that enables tunable tissue-specific gene activation via the splicing modulator risdiplam, and its potential application in myocardial infarction.File in questo prodotto:
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